Jerry R. Mendell, MD

Jerry Mendell

Contact Information


Jerry R. Mendell, MD, is an attending neurologist at Nationwide Children’s Hospital, the Dwight E. Peters and Juanita R. Curran Endowed Chair in Pediatric Research at the Abigail Wexner Research Institute at Nationwide Children’s Hospital and professor of Pediatrics and Neurology at Nationwide Children’s and The Ohio State University.

Dr. Mendell joined the Research Institute at Nationwide Children’s fulltime in 2004, recruited by Philip Johnson, the Head of the Center for Gene Therapy at that time. Shortly after, leadership of the Center changed and provided the opportunity to build and establish a program in Clinical Translation with emphasis on neuromuscular disease. At Nationwide Children’s, he holds academic positions as Professor of Pediatrics and Neurology and holds the Curran-Peters Chair of Pediatric Research.

He is a clinician scientist with decades of experience. A career emphasis began with training in neuromuscular disease at the National Institutes of Health, directing laboratory projects that could be carried to the bedside. He was recruited to Columbus to direct the Neuromuscular Center at Ohio State. His life’s work has emphasized clinical translation. He has published more than 370 articles with a focus on neuromuscular disease and authored books on muscle disease, peripheral nerve disorders, and most recently, gene therapy for muscle disease. He was the principal investigator on the prednisone clinical trial in DMD in 1989 that has profoundly influenced clinical care. He was the PI on the exon skipping trial using eteplirsen, now FDA approved for clinical use in 2016. He was the PI on the first gene therapy trials for DMD and LGMD and was the PI on the SMA gene therapy trial that was FDA approved for systemic delivery for infants with SMA type 1 in 2017. The results of the first systemic gene therapy trial for DMD is now moving to Phase 3. Results from the Phase 1/2 micro-dystrophin trial will be published in June 2020.

Academic and Clinical Areas
Awards, Honors & Organizations
  • Juanita Curran and Dwight Peters Endowed Chair, 2010
  • Distinguished Scholar Award, The Ohio State University, Presented by the President, 2009
  • S. Mouchly Small Scientific Achievement Award, The Muscular Dystrophy Association for Significant Contributions to Neuromuscular Disease Research, 2004
  • Listed, Best Doctors in America

Date of Appointment at Nationwide Children’s Hospital: 08/27/2004

Board Certifications
  • Neurology
Fellowship National Institutes of Health Date Completed: 07/31/1971
Residency National Institutes of Health Date Completed: 06/30/1970
Residency Columbia Presbyterian Medical Center - New York Date Completed: 06/30/1969
Medical School University of Texas Southwestern Medical Center Date Completed: 06/30/1966
Professional Experience

1992 - 2004 Dept of Neurology, The Ohio State University, Chair

Inside Dr. Jerry Mendell's Research Lab

Inside Dr. Jerry Mendell's Research Lab

Inside the Center for Gene Therapy at Nationwide Children’s Hospital is where Dr. Mendell has spent nearly 50 years advancing science for neuromuscular disease through clinical trials. It’s where new treatments and potential life-saving cures are being discovered for families who are racing against the clock for options.